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The FDA this week gave the green light to the first gene therapy for a fatal genetic disorder, as well as a cancer treatment that has never before been given the go-ahead in the US.
The gene therapy, called Lenmeldy, was developed by Orchard Therapeutics and is the first to treat early-onset metachromatic leukodystrophy, a fatal genetic disorder where a mutation in the gene encoding the arylsulfatase A enzyme results in a damaging buildup of sulfatides in cells, particularly in the brain, spinal cord, and peripheral nerves, reports the New York Times.
"The FDA approval of Lenmeldy opens up tremendous new possibilities for children in the US with early-onset MLD who previously had no treatment options beyond supportive and end-of-life care," says a co-founder of Orchard Therapeutics.
The FDA also gave accelerated approval to Bristol Myers Squibb's Breyanzi for the treatment of patients who have had at least two prior lines of therapy for chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL).
To be eligible for this treatment, a patient must have already received at least two prior lines of therapy, including a Bruton tyrosine kinase inhibitor and a B-cell lymphoma 2 inhibitor.